Genethon has entered into an exclusive, worldwide licensing agreement with AskBio to use a patented component of AB-1009 for the treatment of Pompe disease. Pompe disease is a rare genetic disorder caused by a deficiency in the GAA enzyme, leading to glycogen accumulation in cells, particularly in muscle tissue.

The ‘Immunology and Liver Diseases’ team at Genethon, led by Giuseppe Ronzitti, developed and demonstrated the pre-clinical efficacy of a gene therapy approach using a transgene encoding a truncated form of GAA. In animal studies, this approach corrected glycogen accumulation in muscle and the Central Nervous System (CNS) and improved cardiac hypertrophy as well as muscular and respiratory dysfunction. This component of AB-1009, protected by patents held by Genethon and other French institutions, is now licensed to AskBio for the development of a gene therapy product to treat Pompe disease.

This agreement illustrates Genethon’s ability to transform its scientific advances into therapeutic opportunities for patients with rare diseases by collaborating with industry partners to advance clinical development.

“Genethon is delighted with this agreement with AskBio, which enables the development of a gene therapy for patients suffering from this particularly severe condition. This agreement demonstrates the quality of Genethon’s research and its ability to design innovative treatments, driven by its mission to provide therapeutic solutions to patients with rare diseases,” said Frédéric Revah, CEO, Genethon.

AskBio anticipates recruiting its first patient in early 2026.